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Lipid nanoparticle-mediated co-delivery of human RB1 mRNA and triptolide for localized genetic therapy of retinoblastoma

Academic source · Journal of controlled release : official journal of the Controlled Release Society

Source published: 2026-10-09

Site published: 2026-11-03 · Updated: 2026-11-03

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What the paper reports

A C12-200-based formulation was optimized for movement and cellular delivery in the protein-poor vitreous environment. It co-delivered RB1 mRNA and triptolide, improving antiproliferative activity and suppressing tumors in an orthotopic retinoblastoma model after intravitreal administration. Retinal evaluations supported tolerability during the study period. The combination remains preclinical and requires longer ocular safety assessment and pediatric translation studies.

Why this paper is included

Demonstrates tissue-environment-specific engineering for ocular combination therapy; animal tumor suppression and short-term retinal assessments do not establish pediatric clinical safety.

The interpretation above is analysis, not a conclusion attributed to the original source.

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